eligibility_summary
Eligible: ages 3–65 with leukemia/lymphoma/thalassemia/aplastic anemia or other hematologic disease needing allogeneic HSCT, weight 10–100 kg, ECOG ≤3, adequate organs (EF>45%, bilirubin <2×ULN, AST/ALT <3×ULN, creatinine <2×ULN), no severe infection, consent provided. Exclude: non‑hematologic or refusing transplant, life expectancy <1 mo, prior HSCT, pregnancy, severe mental/neurologic issues, or per investigator judgment.
trial_source
clinical_trials.gov from Dec 2, 2025
annotation_status
ai
ai_summary
Intervention: A modified allogeneic HSCT regimen for severe aplastic anemia. Conditioning drugs: • Fludarabine (antimetabolite, purine analog) – lymphodepletion via DNA synthesis inhibition. • Cyclophosphamide (alkylating agent) – DNA crosslinking/cytotoxicity. • Melphalan (alkylating agent) – myeloablation via DNA crosslinking. GVHD prophylaxis: • Post-transplant cyclophosphamide (high-dose alkylator) – selectively depletes proliferating alloreactive T cells. • Antithymocyte globulin, ATG (polyclonal antibody) – T-cell depletion. • Mycophenolate mofetil, MMF (IMPDH inhibitor) – blocks de novo purine synthesis, suppressing T/B-cell proliferation. • Cyclosporine A, CSA (calcineurin inhibitor) – inhibits NFAT/IL-2–mediated T-cell activation. • Ruxolitinib (JAK1/2 inhibitor) – dampens cytokine/JAK-STAT signaling. Targets: recipient immune/hematopoietic cells, alloreactive T cells (Th1/Th17), calcineurin/NFAT, JAK-STAT, and purine synthesis pathways to promote engraftment and reduce GVHD.